Discordance Between Patient and Physician Global Assessments in Early Systemic Sclerosis
Objective This study aims to identify factors associated with patient global assessment (PtGA) and physician global assessment (PhGA) and discordance between them in systemic sclerosis (SSc). Methods Data from adults with early SSc (<5 years) from the Collaborative National Quality and Efficacy Registry were included.
Ellen Romich +35 more
wiley +1 more source
Zilebesiran, a Small Interfering RNA Therapeutic Targeting Angiotensinogen: Mechanism, Clinical Evidence, Safety, and Implementation Considerations. [PDF]
Jawaria +5 more
europepmc +1 more source
Distinct Systemic Sclerosis Phenotypes Related to Ethnicity: An Opportunity to Personalize Care?
Objective The objective is to describe and compare demographic, clinical, and serological characteristics of patients with systemic sclerosis (SSc) according to ethnic background. Methods Participants enrolled in the Canadian Scleroderma Research Group cohort who self‐identified to a single ethnicity group were included.
Danick Goulet +11 more
wiley +1 more source
Toward a Platform PBPK Model to Characterize the Plasma and Tissue Disposition of Unconjugated Antisense Oligonucleotides and GalNAc-Conjugated Small Interfering RNAs in Preclinical Species and Human. [PDF]
Desai DA, Cristofoletti R.
europepmc +1 more source
Objective To evaluate utility of an artificial intelligence (AI) health coach for systemic sclerosis (SSc) self‐management and identify patterns associated with participant engagement. Methods We conducted a mixed methods study in which an AI health coach, powered by a large language model (LLM), was used to support self‐management for SSc.
Nirali Shah +4 more
wiley +1 more source
Application of Dual-Targeting Carbon Dot Nanozymes in Improving Hypoxia and Enhancing Small Interfering RNA-Mediated Gene Silencing for Hepatocellular Carcinoma. [PDF]
Wei Y +9 more
europepmc +1 more source
Caregiver Perspectives on the Burden of Disease and Treatment in Uncontrolled Gout
Objective Uncontrolled gout (UG) refers to persistently elevated serum urate (SU) levels >6 mg/dL and ongoing gout symptoms despite use of urate‐lowering therapy (ULT). The objective of this study was to evaluate the burden associated with informal caregiving for individuals with UG.
Angelo Gaffo +6 more
wiley +1 more source
Safety profile of small interfering RNA therapeutics: a systematic review and meta-analysis of randomized controlled trials. [PDF]
Ahn C +5 more
europepmc +1 more source
Objective To assess the validity of the Patient‐Reported Outcomes Measurement Information System (PROMIS) Pediatric measures in patients with chronic nonbacterial osteomyelitis (CNO). Methods Within the longitudinal patient registry of CNO, English‐speaking patients aged 8 years and older self‐reported PROMIS Pediatric measures of fatigue, pain ...
Mary M. Eckert +43 more
wiley +1 more source
Coding Transcript-Derived Small Interfering RNAs: Their Biogenesis and Molecular Function in Arabidopsis. [PDF]
Xu X +6 more
europepmc +1 more source

